Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)

J. E. Wraith, L. Clarke, Michael Beck et al. · The Journal of Pediatrics · 2004 · 546 citations

Life Sciences, Biochemistry, Enzymology +7

Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome

Susan Staba, Maria L. Escolar, Michele D. Poe et al. · New England Journal of Medicine · 2004 · 481 citations · Full text

Medicine, Hematology, Pediatrics +17

Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure

Jaap Jan Boelens, Robert Wynn, A. O’Meara et al. · Bone Marrow Transplantation · 2007 · 212 citations · Full text

Medicine, Hematology, Surgery +15

Microglia: The effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases

William Krivit · Cell Transplantation · 1995 · 191 citations

Medicine, Neurobiology, Neuroscience +12

Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

Robert Wynn, Jean Mercer, Joanne Page et al. · The Journal of Pediatrics · 2008 · 86 citations

Medicine, Hematology, Surgery +5